Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...2015 Dec 31. pii: aad5725. PubMed PMID: 26721683.
3. Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb... some research in human embryos. Nature. 2015 Dec 3. doi:10.1038/nature.2015.18947
5. Siva K, Covello ... by zinc finger nucleases. Mol Ther. 2015a Mar;23(3):523-32. PubMed PMID: 25492562. PubMed Central PMCID...estimated that very little dystrophin correction (about 4%) is needed to see muscle improvement, with only...diseases we’d like to treat with CRISPR. As seen above, DMD can be treated with “one-size-fits-most” NHEJ-mediated....
Precision genome editing faces the challenges above and more. One chief challenge is upregulating HDR...